CHAPTER 12: Discovery and Therapeutic Relevance of Small Molecule Modifiers of MicroRNA Function

MicroRNAs (miRNAs) are small, non-coding RNAs that predominantly regulate gene expression via translational repression. Dysregulation of miRNAs has been linked to various human diseases, including cancer, suggesting that targeting miRNAs with small molecules may hold therapeutic potential. As such, several approaches have been employed in order to discover small molecule modulators of miRNA function. Here we discuss recent methods used to identify small molecules that target miRNAs. Furthermore, considerations for experimental design as well as investigation into potential modes of action and pre-therapeutic evaluation of miRNA inhibitors are mentioned.